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JR-141 extension study in Mucopolysaccharidosis Type II (Hunter Syndrome)
NCT05594992Phase 3Enrolling by invitation
- Sponsor
- JCR Pharmaceuticals
- Start
- Feb 2023
- Primary completion
- Feb 2028
- Modality
- Replacement Enzymes, Fc-Fusion Proteins
- Countries
- 8
01
Overview
Phase 3, open-label, single-arm trial of pabinafusp alfa in mucopolysaccharidosis type II, sponsored by JCR Pharmaceuticals. Primary endpoints: Heparan Sulfate Concentrations in Cerebrospinal Fluid at 287 weeks, Bayley Scales of Infant and Toddler Development, Third Edition at 287 weeks and 3 more. Started Feb 2023; status: enrolling by invitation.
02
Drugs
pabinafusp alfa
03
Conditions
mucopolysaccharidosis type II
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